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Terisa: My name is Terisa. I have 3 boys. The oldest, Brian, is 14, Tyler is 10, and Benny is 5. [00:00:30] I think that being a mom is probably what my calling's been. I love being their mom. I love being just involved with raising them, and it's what I've always wanted to do. I've always wanted to have kids. I didn't really know what I wanted to do with work. I just knew I wanted my job to be being a mom.

The most rewarding thing is just watching them grow up, just seeing their different personalities, and they're so unique. They all have their own character traits, their own interests. Tyler's hobbies are swimming, playing basketball, [00:01:00] throwing the ball around with his brothers, he likes to do that, likes to cook with me. We like to just hang out and watch movies and videos and he loves video games. That's his favorite thing right there.

When I was pregnant with Tyler, they did a very thorough check, the 18-week fetal survey, just to be proactive since I was born with a heart condition. That's when they found the hole in his heart and some issues, and they diagnosed him prenatally with [00:01:30] Tetralogy of Fallot. He was born at 39 weeks and everything looked good, and they took an X-ray of his heart and that's when I saw the butterfly vertebrae, which is now knowing that that's the first marker that we found for Alagille syndrome. And then, he went home, and we just were looking forward to surgery day and that was it.

He started getting really jaundiced even though he was eating really well. The pediatrician had just run some basic bilirubin tests and he was [00:02:00] really high, but he didn't think it was dangerously high to send him to the hospital. We were going back and forth between cardiology for a week and then pediatrician for a week just to make sure his heart was stable until he was ready for surgery. The morning of pre-op, they just threw on a liver function panel test just to see if the bilirubin dropped.

Next thing you know, surgery's canceled and he is whisked upstairs for a liver ultrasound. Then, that's where they were able to diagnose the bile duct paucity. [00:02:30] They went right from there and clinically diagnosed him with Alagille syndrome.

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When he was diagnosed with Alagille syndrome, I just remember standing there and everything spinning around me. [00:03:00] I just felt like I was in this tornado of like, "What? This just happened to me? I just did everything you told me to do and now I have this diagnosis."

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The light in the tunnel there was finding the Alagille support page and finding people that were going through exactly what I was going through and being able to also connect with families that had the same age child as my kid. It was great to see adults and say, [00:03:30] "Wow, these kids have a future." Ironically, it felt very comforting to have someone on my side.

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Learning about bile acids through the Alagille support page, I realized that that was a big factor to his itch. Basically, your liver has no natural flow of the bile, so it just reabsorbs and it's going through your bloodstream, so everywhere your blood is from head to toe [00:04:00] is going to be itchy. I started noticing him rubbing his eyes a ton. It was more than just the hunger cues, more than the tired cues, and so we went back into another pediatrician and asked, “What's going on?” They said, "No, it's not allergies. He's got to go through a whole season before it's diagnosed as allergies." It was just brushed off as, "We don't know what that is."

The itch was getting worse and every time we did a diaper change, he was scratching at his legs or his diaper area and then rubbing the eyes [00:04:30] constantly. That was the big thing for him. Then, it moved into putting toddler socks on his hands so that they went all the way up to his arms; that way, at night, he wasn't digging into his ears and making them bleed everywhere. The itch kind of normalized for us. We just knew it was there, but at the same time, we really kind of discredited a lot of the itch and a lot of the liver involvement to just being mild, even though we later learned that it was [00:05:00] much more severe than mild.

It just was changing bedsheets consistently, bandaging up all the cuts. Unfortunately, grieving the fact that I couldn't do the bed sharing and the cuddling with Tyler because he sweats so much and then it made him more itchy. Then, trying to sleep with him, he was tossing and turning and rolling and scratching, and I was like, "I can't do this. I'm not sleeping. He's not sleeping." Naps were few and far between. Just was never settled for a nap time because he was so itchy and there was no [00:05:30] medications out there at the time for Tyler to take that actually worked for him to relieve that itch.

Tyler: The itch is just bad. It's horrible and it keeps me from doing stuff. The itch just feels like an itch, a bug bite, or something. But then, it goes from here to your back, and then goes all over, and then you can't stop.

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Terisa: We had learned that LIVMARLI was getting close to being FDA approved and we were very excited about something being an option for him again. We worked with our doctor [00:06:30] and got him on LIVMARLI. Tyler was a good candidate for LIVMARLI because he had Alagille syndrome, but then he had that very persistent pruritus. When he turned 10, he started LIVMARLI and that was great. It was a really fun day for him because it was his birthday, but also it was kind of the start of what could be his new normal, not itching as much as he had been.

On his 10th day was when we started realizing he's sleeping through the night. He's not tossing and turning. [00:07:00] There had been no new scabs, no bandages on his arms and legs. That was a sight to see to not have the bandages so much, but then also just to see him sleeping and his brothers sleeping because they all shared a room and they weren't being woken up by him tossing and turning so much. Once his doctor prescribed LIVMARLI, we got connected with the Mirum Access Plus Program and they got it right on our doorstep in less than 2 months.

It's been nice to not have to be chasing down doctors [00:07:30] for refills and going to the pharmacy to pick things up. It's just so convenient having it right to our door. One side effect that we've been battling is GI cramping. That's just something that we knew it was a possibility of this happening and we had to work with our doctor to make sure that we could manage this for him and let it be so that he could still attend school and do his daily activities. [00:08:00] I think he's great now. I mean, we're going strong on 10 months in. What do you think?

Tyler: I think we're doing good.

Terisa: You feel good on the medicine?

Tyler: Yes.

Terisa: I know 10 years is a long time, but I'm glad he wasn't 20 and he just got his hands on this. I wish it could have been sooner for him, but I'll take 10 years and then the rest of his life.

Tyler: Having Alagille syndrome doesn't really make me feel [00:08:30] much different. It just makes me feel like myself because it doesn't affect what's on the outside. It mostly affects what's on the inside. Now that I'm on LIVMARLI, I'm itching less and I'm sleeping better.

Terisa: There's people out there that are willing to connect and happy to connect and make sure that you feel supported through your journey because at the beginning, it can be very overwhelming and you can feel like you're drowning, but there's light on the other side and there's [00:09:00] a possibility of having happiness as part of your every day.

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Narrator:

INDICATION

LIVMARLI is a prescription medicine used to treat cholestatic pruritus (itch) in patients with Alagille syndrome 3 months of age and older. It is not known if LIVMARLI is safe and effective in children under 3 months of age or in adults 65 years and older.

IMPORTANT SAFETY INFORMATION

What are the possible side effects of LIVMARLI? [00:09:30]

LIVMARLI can cause serious side effects, including:

Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome but may worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your health care provider should do blood tests before starting and during treatment to check your liver function. Tell your health care provider right away if you get any signs or symptoms of liver problems, including:

• nausea or vomiting
• skin or the white part of the eye turns yellow
• dark or [00:10:00] brown urine
• pain on the right side of the stomach (abdomen)
• loss of appetite

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your health care provider right away if you have any of these symptoms more often or more severely than normal for you.

A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamins A, D, E, and K) stored in body fat. [00:10:30] FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your health care provider should do blood tests before starting and during treatment.

Other common side effects reported during treatment were gastrointestinal bleeding and bone fractures.

Tell your health care provider about all medicines that you take. LIVMARLI may affect the way some other medicines work, and some other medicines may affect the way LIVMARLI works. If you take a medicine that lowers cholesterol by binding bile acids, such as cholestyramine, [00:11:00] colesevelam, or colestipol, take it at least 4 hours before or 4 hours after you take LIVMARLI.

LIVMARLI is taken by mouth, 1 time each day, 30 minutes before a meal in the morning. Be sure to use the provided oral dosing dispenser to accurately measure the dose of medicine.

These are not all of the possible side effects of LIVMARLI. Call your health care provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

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